Aduhelm: What the Discontinued Alzheimer’s Medication Reveals About the Drug Approval Process

Image Citation: [1]

Written by Grace Li ‘26

Edited by Yumiko Imai ‘26

For the 6.7 million Americans grappling with Alzheimer’s, the lack of effective treatments has long been a source of despair [2]. Existing drugs offer only temporary relief from symptoms, leaving the underlying disease largely unaddressed. Aduhelm, Biogen’s brand-name drug for aducanumab, was approved by the FDA in 2021 as the first new therapeutic approach for Alzheimer’s in two decades [3]. It stands as the only emerging option aimed at modifying the disease’s underlying pathology. Thus, for the millions of Americans living with Alzheimer's and their caregivers, Aduhelm wasn’t merely a drug—it was a symbol of hope, a chance to cling to their memories and continue participating in life’s precious milestones.

However, Aduhelm's story is not as much about patient needs as it is about corporate coercion and regulatory failure. The drug’s tumultuous journey to approval sheds light on the deep divisions within the regulatory landscape, raising important questions about standards, scientific integrity, and the tension between the urgent desire for hope and the need for solid evidence.

The drug’s development was initially halted by its maker Biogen in 2019 when late-stage clinical trial data suggested it did not meet its efficacy endpoints [3]. With trials discontinued, many in the scientific community dismissed Aduhelm as another failed Alzheimer’s candidate in a long line of disappointments. Despite this, Biogen revisited the data and found new evidence indicating the drug could clear beta-amyloid plaques in the brain—protein aggregates believed to contribute to the progression of Alzheimer’s disease [3]. This led to their decision to re-submit Aduhelm for regulatory approval.

The FDA ultimately granted Aduhelm accelerated approval in 2021, a pathway designed for drugs that addressed unmet medical needs, based on the surrogate endpoint of amyloid plaque reduction. However, whether this reduction translates into meaningful cognitive benefits remains unproven. This made the approval highly controversial. In fact, the FDA’s decision went against the recommendation of its own advisory committee, which unanimously opposed the drug’s approval [3]. The committee, which included an expert group of neurologists and scientists, argued that the available evidence was insufficient to prove that the drug's plaque-clearing effects actually benefit patients [3]. While some believed the decision to grant Aduhelm accelerated approval was driven by the desperate need for a breakthrough Alzheimer’s treatment, others argued that it represents a dangerous precedent in regulatory practice where scientific uncertainty is overridden by the desire for hope.

The reality following this approval, however, was far from idealistic. Biogen has priced Aduhelm at an astonishing $56,000 per year—a strategic move that has driven its stock price up by more than 50%, reaching its highest closing level in over six years [4]. This exorbitant cost makes the treatment unaffordable for most patients paying out of pocket. Meanwhile, Medicare, the public insurance that most Alzheimer’s patients are enrolled under, has restricted coverage to clinical trial participants, citing serious safety risks and uncertain benefits [5]. As a result, the vast majority of patients remain unable to access the drug.

In 2022, a congressional investigation revealed excessive collaboration between the FDA and Biogen, a lack of proper documentation, and an unusual joint presentation to the advisory committee that did not follow standard procedure [6]. Finally, in January of 2024, Biogen decided to pull Aduhelm out of the market, marking the end of the story of what was once dubbed as history’s greatest blockbuster Alzheimer’s drug [7].

Ultimately, Aduhelm’s whirlwind tour from approval to retraction provided little to no direct benefit to patients. The true legacy of Aduhelm lies in its regulatory failures, demonstrating how opening the door for drugs with questionable efficacy sets a dangerous precedent. This not only risks crowding out truly effective treatments but also threatens the credibility of the entire approval process, leading to payer withdrawal and physician rejection. Aduhelm’s saga stands as a stark alarm bell, warning of the consequences when regulatory oversight falters.

References

1. Nania R. Aduhelm Gets Pulled From the Market [Internet]. AARP. 2024. Available from: https://www.aarp.org/health/drugs-supplements/info-2024/aduhelm-alzheimers-drug-taken-off-market.html

2. Alzheimer's Association. 2023 Alzheimer’s Disease Facts and Figures. Alzheimer’s & Dementia [Internet]. 2023 Mar 14;19(4):1598–695. Available from: https://pubmed.ncbi.nlm.nih.gov/36918389/

3. Belluck P, Kaplan S, Robbins R. How an Unproven Alzheimer’s Drug Got Approved. The New York Times [Internet]. 2021 Jul 20; Available from: https://www.nytimes.com/2021/07/19/health/alzheimers-drug-aduhelm-fda.html

4. Steenhuysen J, Beasley D. U.S. approval of Biogen Alzheimer’s drug sends shares soaring, hailed as 'big day" for patients. Reuters [Internet]. 2021 Jun 8; Available from: https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-set-rule-controversial-biogen-alzheimers-drug-2021-06-07/

5. Belluck P. Medicare Officially Limits Coverage of Aduhelm to Patients in Clinical Trials. The New York Times [Internet]. 2022 Apr 7; Available from: https://www.nytimes.com/2022/04/07/health/aduhelm-medicare-alzheimers.html

6. Belluck P. Congressional Inquiry into Alzheimer’s Drug Faults Its Maker and F.D.A. The New York Times [Internet]. 2022 Dec 29; Available from: https://www.nytimes.com/2022/12/29/health/alzheimers-drug-aduhelm-biogen.html

7. Christensen CH Jen. Biogen discontinues Alzheimer’s medication Aduhelm [Internet]. CNN. 2024. Available from: https://www.cnn.com/2024/01/31/health/aduhelm-alzheimers-biogen/index.html